FDA grants rare pediatric disease designation to treatment for systemic JIA flares
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The FDA has granted rare pediatric disease designation to a type IA prodrug of dexamethasone that targets CD206+ macrophages for the treatment of systemic juvenile idiopathic arthritis flares, according to a press release from PIF Partners.
The proprietary investigational therapeutic, currently known as 101-PGC-005 or ‘005 (PIF Partners), is undergoing phase 3 clinical trials across nine centers in India for the treatment of autoimmune rheumatic diseases induced by COVID-19. Under the rare pediatric disease designation, PIF Partners is eligible to receive a priority review voucher upon approval, which can be used to expedite review on another product or sold to another sponsor.
“We are thrilled the FDA has recognized the potential of ‘005 to address the significant unmet medical needs of children suffering from [systemic JIA],” Alec Goldberg, CEO of PIF Partners, said in the release. “This designation is a pivotal milestone that reinforces our commitment to developing innovative therapies for rare pediatric inflammatory diseases. By targeting activated macrophages with the most powerful anti-inflammatory compounds, we enhance efficacy and reduce toxicity.
“Critically, we have demonstrated no suppression of the [hypothalamic-pituitary-adrenal] system, thus supplementing rather than replacing the natural anti-inflammatory response,” Goldberg added. “We are eager to advance our clinical program and expand our research into other orphan diseases characterized by systemic inflammation.”
In addition to 101-PGC-005, PIF Partners’ pipeline consists of other exclusively licensed drug candidates that target CD206+ macrophages, according to the release. The company is looking for a partner to “rapidly progress the development and commercialization” of new therapies for rare diseases of systemic inflammation.
Earlier this year, the FDA approved Rinvoq (upadacitinib, AbbVie) for active, polyarticular JIA, as well as an oral form of methotrexate, Jylamvo (Shorla Oncology), for polyarticular JIA.
Under current law, the rare pediatric disease priority review voucher program is scheduled to sunset after Dec. 20.
References:
Rare pediatric disease designation and priority review voucher programs. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs. Updated Sept. 27, 2024. Accessed Dec. 4, 2024.
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